Gene therapy works in mice to prevent blindness that strikes boys

Researchers injected a healthy version of the human RS1 gene to the sub-retinal space of the right eyes of 15-day-old male mice, which, like boys with the disease, don't have the healthy gene to maintain the retina. In terms of disease development, the condition in the mice was roughly equivalent to retinoschisis in a 10-year-old boy. Six months later, researchers looked at the interior of the eyes with a laser ophthalmoscope and found cyst formation was clearly evident in the untreated eyes, but the treated eyes appeared healthy. The eye's photoreceptor cells - the rods and cones that help the brain process light and color - were spared from the disease and the connections between the layers of the retinas were intact.

Gene therapy works in mice to prevent blindness that strikes boys

1 comment:

Neil said...

Fantastic. The application of this might not be limited to young people. Maybe one day soon, there will be adults who will greatly benefit from this research.

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